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Prader-Willi SyndromeNCT00372125

Nordic Study on the Effects of Growth Hormone (Norditropin SimpleXx) Treatment in Adults With Prader-Willi Syndrome

A study testing Norditropin SimpleXx in people with Prader-Willi Syndrome, run by Karolinska University Hospital at 3 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The aim of this study is to study the effects of GH on body composition, lipid and glucose metabolism, physical performance and safety aspects in adults with PWS.The patients are randomized to either GH or placebo the first year of the study, subsequently followed by two years of GH treatment. Read more about this study

Who is it for?
Ages 18 to 50 years
Conditions the study is for
Prader-Willi Syndrome
What is being tested?
Norditropin SimpleXx
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
46 participants
Where
3 sitesin 3 countries
Study dates
Apr 2005to Mar 2010
Phase
Interventional
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 50 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • Genetically verified PWS diagnosis (by methylation and FISH test.)
  • Between 18 and 50 years old
  • Informed consent obtained before any trial-related activities. (Trial-related activities are any procedure that would not have been performed during normal management of the treatment of the subject.)

Who cannot join

  • Known or suspected allergy to GH preparation.
  • Previous participation in this trial.
  • GH treatment within the last 1 years
  • Malignancy or other serious diseases (ex severe cardiovascular diseases, severe infections)
  • Sexhormone treatment initiated within the last year
  • Pregnancy
  • Untreated respiratory impairment, untreated sleep apnoea or untreated respiratory infection.

Trial locations and contacts

3 sites in 3 countries.

3 sites in 3 countries

Denmark1 site

  • Center for rare Diseases, Department of Pediatrics, Skejby University Hospital

    Aarhus N, 8200

Other trials in Denmark

Norway1 site

  • Endokrinologisk seksjon, Med Avd, Rikshospitalet

    Oslo

Other trials in Norway

Sweden1 site

  • Department of Endocrinology and Diabetology, Karolinska Hospital

    Stockholm, 171 76

Other trials in Sweden

About this study

The aim of this study is to study the effects of GH on body composition, lipid and glucose metabolism, physical performance and safety aspects in adults with PWS.The patients are randomized to either GH or placebo the first year of the study, subsequently followed by two years of GH treatment. the study is performed in Norway, Sweden and Denmark.

Read the detailed description

OBJECTIVE(S):

Prader Willi syndrome (PWS) is a multi-symptomatic genetic disorder associated with abnormalities in the growth hormone (GH)-insulin-like-growth factor (IGF)-I axis and in the body composition. GH treatment is a registered indication in children with PWS, and improves growth rate and body composition. One pilot study in adult patients with clinical PWS has shown beneficial effects on body composition without simultaneous significant side effects. The aim of the present study is to evaluate the effects of GH treatment on body composition, muscle function and quality of life in PWS adults.

TRIAL DESIGN:

The study will be an investigator initiated and investigator sponsored multinational and multi-centre trial, including centres in Norway, Sweden and Denmark. Within each centre patients will be randomised (double blind) to one year treatment with daily injections of GH or placebo (efficacy), followed by a two year observation period on GH treatment (safety).

TRIAL POPULATION:

Twenty patients from each centre are included in the study. The patients need a genetically verified diagnosis and should be between 18 and 40 years old. Patients are excluded if GH treatment has been given within the last two years, if they have a malignancy or other serious diseases, in particular severe respiratory diseases.

ASSESSMENTS:

Effect is evaluated primarily as changes in body composition, activity of daily living and quality of life.

SAFETY: Before starting in the study all patients will be examined for tonsillary hypertrophy and sleep apnoea. Oral Glucose Tolerance Tests will be performed regularly.

TRIAL PRODUCT(S):

During the initial 4 weeks of the placebo-controlled study phase patients will be treated with sc injections of GH (Norditropin Simplexx) in the evening with doses of 0.3 mg/day respectively 0.4 mg/day if BW is below or above 100 kg. Thereafter doses will be increased to 0.6 mg/day (0.8 mg/day) and maintained fixed for 11 months. During the following 24 months open phase doses will be individually titrated.

Being tested
Norditropin SimpleXx
Keywords
Prader-Willi syndrome, Growth hormone, Body composition
Medical subject headings
Prader-Willi Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Norditropin SimpleXxDrug

    0.3-0.4 mg/day placebo or GH for 4 weeks. Thereafter 0.6-0.8 mg/day placebo or GH for 11 months. During the following 24 months open phase doses will be individually titrated.

Study groups in NCT00372125
Study groupRoleWhat they receive
Norditropin SimpleXxActive comparator0.3 mg/day or 0.4 mg/day if bodyweight was below or above 100 kg,for 4 weeks, 0.6 mg/day or 0.8 mg/day, for 11 months.
Receives: Drug: Norditropin SimpleXx
PlaceboPlacebo comparatorPlacebo for 12 months
Receives: Drug: Norditropin SimpleXx

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Changes in body composition (lean body mass and fat mass) measured by dual energy X-ray absorptiometry (DXA)

    Measured: 36 months

Secondary outcomes (10)

  1. Bone mineral density measured by DXA

    Measured: 36 months

  2. Effects on forced expiratory volume (Peakflow)

    Measured: 36 months

  3. Standard photography appearance according to visual analogue scale (VAS)

    Measured: 36 months

  4. Effects on free and total IGF-I, IGF-binding protein (BP)-1 and 3

    Measured: 36 months

  5. Effects on lipids (fasting triglycerides(TG), total, HDL and LDL cholesterol)

    Measured: 36 months

  6. Effects on body composition measured with bioimpedance

    Measured: 36 months

  7. Effects on haemoglobin (Hb), leucocyte and thrombocyte counts, FSH, LH, estradiol, Testosterone, inhibin B, TSH and Thyroxine

    Measured: 36 months

  8. Muscle and fat mass measured by abdominal and mid-femoral computerized tomography

    Measured: 36 months

  9. Activity of daily living measured a.m. Guralnik

    Measured: 36 months

  10. Quality of life estimated by questionnaires

    Measured: 36 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Sep 2006 (estimated)
  2. Study startApr 2005
  3. Primary completionMar 2010
  4. Study completionMar 2010

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase N/A
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
46 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00372125
Sponsor's protocol ID
CH1234
Status last verified
Sep 2016
First submitted
5 Sep 2006
Last updated
13 Sep 2016
Results posted
No
Data monitoring committee
Yes

Publications

  • Shukur HH, Hussain-Alkhateeb L, Farholt S, Norregaard O, Jorgensen AP, Hoybye C. Effects of Growth Hormone Treatment on Sleep-Related Parameters in Adults With Prader-Willi Syndrome. J Clin Endocrinol Metab. 2021 Aug 18;106(9):e3634-e3643. doi: 10.1210/clinem/dgab300.

    PubMed 33950234

  • Olarescu NC, Jorgensen AP, Godang K, Jurik AG, Froslie KF, Bollerslev J. Dual-energy X-ray absorptiometry is a valid method to estimate visceral adipose tissue in adult patients with Prader-Willi syndrome during treatment with growth hormone. J Clin Endocrinol Metab. 2014 Sep;99(9):E1727-31. doi: 10.1210/jc.2014-2059. Epub 2014 Jun 23.

    PubMed 24955611

  • Jorgensen AP, Ueland T, Sode-Carlsen R, Schreiner T, Rabben KF, Farholt S, Hoybye C, Christiansen JS, Bollerslev J. Glucose homeostasis in adults with Prader-Willi syndrome during treatment with growth hormone: results from a 12-month prospective study. Growth Horm IGF Res. 2014 Feb;24(1):16-21. doi: 10.1016/j.ghir.2013.11.002. Epub 2013 Dec 4.

    PubMed 24360789

  • Jorgensen AP, Ueland T, Sode-Carlsen R, Schreiner T, Rabben KF, Farholt S, Hoybye C, Christiansen JS, Bollerslev J. Two years of growth hormone treatment in adults with Prader-Willi syndrome do not improve the low BMD. J Clin Endocrinol Metab. 2013 Apr;98(4):E753-60. doi: 10.1210/jc.2012-3378. Epub 2013 Feb 22.

    PubMed 23436915

Questions about this trial

What is this clinical trial studying?

A study testing Norditropin SimpleXx in people with Prader-Willi Syndrome, run by Karolinska University Hospital at 3 sites in 3 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 13 Sep 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in Denmark, Norway, Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Changes in body composition (lean body mass and fat mass) measured by dual energy X-ray absorptiometry (DXA) (time frame: 36 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00372125, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Sep 2016.

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